Paediatric-first oncology company whose tovorafenib became the first targeted therapy approved for childhood low-grade glioma driven by BRAF fusions, following dabrafenib with trametinib (2023) for BRAF V600E tumours.
Day One Biopharmaceuticals, based in Brisbane, California, and listed as DAWN, is a paediatric-first oncology company whose tovorafenib became the first targeted therapy approved for childhood low-grade glioma driven by BRAF fusions, following dabrafenib with trametinib (2023) for BRAF V600E tumours. Ojemda, its brand of tovorafenib, was approved in the United States in 2024 on the FIREFLY-1 trial, Ipsen holds the rights outside the United States, the LOGGIPY-2 phase 3 tests it in the first-line setting, and pimasertib, a MEK inhibitor, is being developed in combinations. OnCo links it to glioma and paediatric low-grade glioma and to the bottleneck of rare and paediatric cancers without markets, which its model of developing drugs for children first is meant to answer. Whether a company can sustain itself on paediatric indications alone is the open question. Tovorafenib has its own page.
Tovorafenib is a pill for the most common childhood brain tumour, low-grade glioma driven by BRAF changes, approved in 2024.
Shares FIREFLY-1, Paediatric low-grade glioma.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets.
Shares FIREFLY-1, Tovorafenib, Paediatric low-grade glioma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets.
Shares Tovorafenib, Paediatric low-grade glioma.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets, Glioma & glioblastoma.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets.
Shares Tovorafenib, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Rare and paediatric cancers without markets, Glioma & glioblastoma.